In 2006, Marty was diagnosed with myelofibrosis during an annual physical and was told he had three to five years to live. Marty is the epitome of a powerful patient – when diagnoses, he turned to the internet to research his condition. He read about Dr. Verstovsek and the clinical trials he was conducting. Marty eventually met him at a conference, became Dr. Verstovsek’s patients, and enrolled in the JAK2 clinical trial.

Dr. Verstovsek explains the JAK2 trial and Marty’s participation. Dr. Verstovsek addresses how to determine the best treatment approach, considering that many myeloproliferative disorders have a “watch and wait” period. He stresses the importance of advances in research, how they are providing hope for the future. Dr. Verstovsek encourages listeners to participate in clinical trials. Listen now to learn more about myeloproliferative disorders.

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Myeloproliferative disorders can strike at any age, and they have no known cause. Symptoms, prognosis and progression vary. Some patients may have slowly progressing disease that may not require any treatment beyond careful monitoring while others may progress to more advanced stages, or even in some cases, AML, acute myelogenous leukemia. We'll learn all about this next on Patient Power.

Hello and welcome once again to Patient Power. I'm Andrew Schorr. I want to tell you in this broadcast about myeloproliferative disorders. They can strike at any age. They have no known cause, but at M. D. Anderson there is some encouraging research, and so it's very important to get to a specialty center like M. D. Anderson, because depending upon where you are the doctors may not be knowledgeable, and we're talking about rare conditions, and we're talking about a variety of conditions, but there is encouraging research, the latest genetic research and news coming out of that. We're going to hear all about that in our program.

And as we like to do, we like to introduce you to a patient who has really lived this story. So let me introduce you to Marty Prager, who joins us from Burbank, California. Marty is 60 years old, almost 61. Married, two kids, and he is in the motion picture business. And then, Marty, you go for an annual physical, 2006 you go for an annual physical, and what did the doctor say from the routine blood test?

Marty:

I had my physical in 2006, and my physician said that I had myelofibrosis and said I had three to five years to live.

Andrew Schorr:

Oh, my goodness. Now, this is something you never heard of, and life threatening condition. So one of the doctors, I know, and you were in an HMO, says, well, do your research, get on the internet, learn all you can. And when you did you connected with an advocacy group that had information, and you even saw there was a conference coming up of all the leading doctors, right?

Marty:

That's correct.

Andrew Schorr:

So you said, I'm going.

Marty:

That's right. That's what I did.

Andrew Schorr:

So this is really a powerful patient taking the bull by the horns. Were you just feeling in Los Angeles that you just weren't encountering an expert, and you had a concern that whatever was the latest information could make a difference?

Marty:

Well, it's such a rare disease that I felt that a gathering of the acknowledged experts in the field is where I should be, and that's why I went to Scottsdale. It's not that far away. It seemed like the logical thing to do.

Andrew Schorr:

So this is a conference put on by the CMPD Education Foundation, right?

Marty:

Yes. That's correct.

Andrew Schorr:

All right. So you go, but before you go you hear that one of the speakers there and one of the leading experts is Dr. Srdan Verstovsek, who is a medical oncologist and associate professor in the leukemia department at M. D. Anderson, an expert in this. You actually got in touch with him, right?

Marty:

Yes. We exchanged a couple of phone calls and a couple of e mails.

Andrew Schorr:

So you said I got to meet this guy, and so he gives a speech at this conference and you come up to him afterwards, and what do you begin to talk about as far as research that could make a difference?

Marty:

Well, I explained that I understood that he was conducting a clinical trial and the trial was due to start soon, and I honestly didn't know how to approach someone about participating in a clinical trial, so I just introduced myself and told Dr. V. that I was interested in the clinical trial, and would I be suitable for the trial.

Andrew Schorr:

Now, at this same time as time was going on, you're getting transfusions because your anemia is quite significant. I think normal hemoglobin is about 14, and you had dropped to seven, right?

Marty:

That's right.

Andrew Schorr:

But you're still trying to work, so you're getting transfusions every three weeks to kind of pump you up, but even so you had what was believed to be a life threatening condition.

Marty:

Yes. That's correct.

Andrew Schorr:

Yeah, that's no way to live. Okay. Well, you mentioned Dr. V, so again, that's Dr. Srdan Verstovsek, and this is his field.

Dr. V, so this is a wonderfully proactive patient who approaches you, and it's at a time when now finally there is some promising research. What was going on in the research world? Was this out of the human genome project, or what was happening, where for a rare condition you might have something new.

Dr. Verstovsek:

Yes, this is the beginning of 2007, and a couple of years earlier, in earlier 2005 there was a major discovery made in the field of myeloproliferative disorders, discovery of a mutation in a gene called J A K 2, or JAK2, which makes the protein, and the mutated JAK2 protein appears to be contributing to the continuous unregulated growth of cells in these myeloproliferative diseases. So at the conference in 2007 we were talking about future studies with the specific targeted therapy, JAK2 inhibitors that were supposed to inhibit that mutated JAK2 protein and hopefully help the patients, and Martin is one of the patients participating in these early studies.